Eplontersen is a triantennary N-acetyl galactosamine (GalNAc3-7a) conjugated antisense oligonucleotide that selectively targets transthyretin (TTR) mRNA. By inhibiting the synthesis of both variant and wild-type TTR proteins, Eplontersen plays a crucial role in disrupting the misfolding of TTR, which contributes to the formation of amyloid fibrils associated with ATTR amyloidosis. This compound is valuable for research applications focused on amyloid-related diseases and exploring therapeutic strategies for TTR-related pathologies.
Eplontersen is a triantennary N-acetyl galactosamine (GalNAc3-7a) conjugated antisense oligonucleotide that selectively targets transthyretin (TTR) mRNA. By inhibiting the synthesis of both variant and wild-type TTR proteins, Eplontersen plays a crucial role in disrupting the misfolding of TTR, which contributes to the formation of amyloid fibrils associated with ATTR amyloidosis. This compound is valuable for research applications focused on amyloid-related diseases and exploring therapeutic strategies for TTR-related pathologies.
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